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Search Results Within Category "Diabetes/Endocrinology/Metabolism"

Here are the studies that match your search criteria. If you are interested in participating, please reach out to the contact listed for the study. If no contact is listed, contact us and we'll help you find the right person.

15 Study Matches

Liraglutide Treatment in Patients With Maturity-onset Diabetes of the Young (MODY)

The reason for this research study is to better understand the use of liraglutide, a commonly prescribed Type 2 diabetes medication, in patients with a diagnosis of maturity-onset diabetes of the young (MODY). The investigators are interested in better understanding the way that this drug affects the metabolism and hormone levels of a person with MODY. Many people with MODY report having gastrointestinal (GI) issues such as an upset stomach. Investigators also are interested in finding out if this drug will help with GI issues. If liraglutide does help with this symptom of MODY, the investigators want to know why this happens. If this drug is effective for participants, the investigators will use participants cells to make human induced pluripotent stem cell (iPSC). This means that the investigators will use participant cells to create what are called stem cells, which are cells in the body that are able to be told what their job is. Investigators will use these cells to see what happens in gastrointestinal (GI) tract.

Lily Deng - lily.deng@cchmc.org

ALL
10 years and over
PHASE2
This study is NOT accepting healthy volunteers
NCT07029009
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Inclusion Criteria:
* Diagnosis of MODY via genetic testing * HgbA1c \>6.5%
Exclusion Criteria:
* History of anaphylaxis to GLP-1 receptor agonists * Already taking GLP-1 receptor agonists * on medications within the stimulant class * have had bariatric surgery * personal or family history of cancer, especially medullary thyroid cancer * personal history of pancreatitis or pathogenic variants associated with increased risk of pancreatitis * known active kidney disease * severe hypoglycemia (glucose \< 50 mg/dL) within 3 months of study enrollment * HbA1c \> 10.0% * episode of diabetic ketoacidosis (DKA) in the past 3 months * are currently pregnant * BMI \< 18.5 kg/m2 and pediatric patients with BMI \< 5th percentile
DRUG: Liraglutide
Maturity Onset Diabetes of the Young
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A Study to Assess Growth in Children With Idiopathic Short Stature

Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.

Cierra Farrell - cierra.farrell@cchmc.org

ALL
2 years to 16 years old
This study is NOT accepting healthy volunteers
NCT06309979
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Inclusion Criteria:

• Participants must be \> 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.
• A height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth chart (https://www.cdc.gov/growthcharts/zscore.htm).
• Participants who have either never received hGH, or who are currently receiving hGH treatment.
• Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.
Exclusion Criteria:

• Diagnosis of systemic disease or condition that may cause short stature, eg renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. Children with such diagnoses can be considered for inclusion if their condition is well controlled, at the discretion of the Medical Monitor.
• Known presence of one or more pituitary hormone deficiencies
• Bone age advanced over chronological age by more than 3 years.
• For hGH naïve participants, historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (between -1.00 SDs and +2.00 SDs).
• For participants currently on hGH treatment, historic results before GH treatment of stimulation test with serum or plasma GH level greater than 10 μg/L or serum IGF-1 test between -1.00 SDs and +2.00 SDs.
• Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .
Idiopathic Short Stature
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DISCOVERY of Risk Factors for Type 2 Diabetes in Youth (DISCOVERY)

The goal of the DISCOVERY study is to provide innovative critical information regarding the unique natural history of glycemic control, insulin sensitivity, and β-cell function, and their mechanistic determinates, in obese adolescents at risk for developing type 2 diabetes.

Hailey Eisenmann - discoveryinteens@cchmc.org

ALL
8 years to 15 years old
NCT06525259
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Inclusion Criteria:
Screening will occur to enrich the yield of individuals who are highly predisposed to develop youth-onset T2D and include those with all of the following criteria: * Overweight or obesity with BMI ≥85th percentile * Age 8-13 year for girls, 9-15 year for boys (inclusion younger for girls as puberty tends to start a year earlier in girls) * Tanner Stage 2, 3, or 4 * Elevated HbA1c 5.5-6.4%
Exclusion Criteria:
An individual who meets any of the following criteria will be excluded from participation: * Diabetes based on history, or HbA1c ≥6.5% in the medical record or at screening * Unable/unwilling to provide consent/participate fully * Conditions predisposing to diabetes or altering the trajectory of puberty * Medications affecting glucose dynamics during the screening and enrollment period * Prior treatment with insulin * Use of glucagon-like peptide-1 (GLP-1) receptor agonist or any weight loss medications in the 6 weeks prior to enrollment * Planning treatment with glucagon-like peptide-1 (GLP-1) receptor agonist or any weight loss medications * Use of metformin or any glucose lowering medication for a reason other than treatment of diabetes (e.g., for PCOS) in the 6 weeks prior to enrollment * Planning treatment with metformin or any glucose lowering medication or planning to use such medications for a reason other than treatment of diabetes (e.g., for PCOS) * Known syndromic/monogenic obesity * Blood disorders impacting HbA1c (e.g., anemia, hemoglobin variants) * Major systemic organ disease * History of bariatric surgery or currently planning bariatric surgery * Current pregnancy or currently planning pregnancy * Use of GnRH agonist, estrogen, or testosterone * Individuals who do not speak English or Spanish, given validation of the questionnaires to be utilized
Diabetes Mellitus Type 2, Childhood-Onset
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Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD)

The purpose of this study is to evaluate the safety, pharmacodynamics (PD), and exploratory clinical efficacy of S-606001 in adult participants with LOPD as an add-on to ERT.

Laurie Bailey - Laurie.Bailey@cchmc.org

ALL
18 years and over
PHASE2
This study is NOT accepting healthy volunteers
NCT07123155
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Key
Inclusion Criteria:
* Participant must be ≥18 years of age and ≥40 kilograms (kg) of body weight at the time of signing the informed consent. * Participant must have a diagnosis of LOPD based on documentation of 1 of the following:
• Deficiency of acid alpha-glucosidase (GAA) enzyme
• GAA genotype * Participant has a %FVC ≥30% and ≤80% in an upright position without mechanical ventilation at screening; or Participant has ≥10% %FVC drop from upright position to supine position and %FVC ≥20% in a supine position. * Participant performs the 6MWT at screening, as determined by the clinical evaluator, and meets all of the following criteria:
• Screening values of 6-minute walk distance (6MWD) are ≥75 meters
• Screening values of 6MWD are ≤90% of the predicted value for healthy adults * Participants must be ERT-experienced, defined as currently receiving ERT and having been receiving ERT for ≥24 months, with no regimen change in the last 6 months. Key
Exclusion Criteria:
* Has a medical condition or any other extenuating circumstance that may pose an undue safety risk to the participant or may compromise his/her ability to comply with or adversely impact protocol requirements. * Has active infections at screening. * Malignancy within the past 5 years except for basal cell or squamous epithelial carcinomas of the skin that have been resected with no evidence of metastatic disease for 3 years. * Current or chronic history of liver disease. * Known biallelic loss of function mutations whether in glycogenin gene (GYG) or in glycogen phosphorylase muscle associated gene(PYGM) . * Has received any investigational therapy or pharmacological treatment for Pompe disease, within 30 days or 5 half-lives of the therapy or treatment, whichever is longer, before day 1 or is anticipated to do so during the study. * Has received gene therapy or small interfering ribonucleic acid (RNA) therapy for Pompe disease. * Participant, if female, is pregnant or breastfeeding at screening. * Participant, whether male or female, is planning to conceive a child during the study. Note: Other protocol-specified inclusion and exclusion criteria may apply.
DRUG: S-606001, DRUG: Placebo
Pompe Disease
Late-onset Pompe disease, Enzyme replacement therapy, LOPD, ERT, S-606001, Muscle glycogen synthase, Liver glycogen synthase, Rare disease, Autosomal disease, Acid alpha-glucosidase, GAA, Glycogen storage disorder, GSD
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Global Patient Registry to Monitor Long-term Safety and Effectiveness of Increlex® in Children and Adolescents With Severe Primary Insulin-like Growth Factor-1 Deficiency (SPIGFD).

The Increlex® Global Registry is a descriptive, multicenter, observational, prospective, open-ended, non interventional, post-authorisation surveillance registry. The main purpose of this global registry is to collect, analyse and report safety data during and up to at least 5 years after the end of treatment in children and adolescents receiving Increlex® therapy for SPIGFD according to the locally approved product information.

Leah Tyzinski - Leah.Tyzinski@cchmc.org

ALL
2 years to 18 years old
This study is NOT accepting healthy volunteers
NCT00903110
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Inclusion Criteria:
* For US : patients starting or planning to start or currently receiving treatment with Increlex® therapy for severe primary IGF-1 deficiency as defined by the US Increlex® prescribing information or for growth hormone (GH) gene deletion who have developed neutralizing antibodies to GH.For EU : patients starting or planning to start or currently receiving treatment with Increlex® therapy according to the locally approved product information. * Parents or legally authorized representatives if applicable must give signed informed consent before any registry-related activities are conducted. Assent from the subject should also be obtained as appropriate
Exclusion Criteria:
* Subject currently participating in an Increlex® clinical trial * Subject currently participating in any clinical trial for growth retardation * Patient with any contraindication to Increlex® or any condition subject to special warning as per the locally approved label * For US patients, these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia and patients with closed epiphyses. * For EU patients: these include patients with hypersensitivity to the active substance or any of the excipients, patients with active or suspected neoplasia or any condition or medical history which increases the risk of benign or malignant neoplasia and patients with closed epiphyses
DRUG: Increlex®
IGF1 Deficiency
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Effects of Exercise on Lipids and Endothelial Function in Youth

This study is for individuals who have a BMI that is at or above the 95th percentile and are participating in the Cincinnati Children's Hospital Healthworks! Structured weight loss program. The main reason for this research study is to determine how exercise affects an individual's high level of lipids (fats) in their blood and how well their blood vessels function.

Erin Cain - erin.cain@cchmc.org

ALL
10 years to 20 years old
NA
This study is NOT accepting healthy volunteers
NCT07409064
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Inclusion Criteria:

• Age: 10-20 years
• Obesity as defined by BMI ≥ 30 kg/m2 OR BMI ≥ 95%ile for age and sex
• Planned to start the Healthworks! structured weight loss program.
• English or Spanish speaking -
Exclusion Criteria:
1\. Physician judgement about inability to finish the protocol \-
OTHER: Exercise Program
Obesity & Overweight
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A Global Prospective Observational Registry of Patients With Pompe Disease

This is a global, multicenter, prospective, observational registry of patients with Pompe disease, including those with late-onset pompe disease (LOPD) and infantile-onset pompe disease (IOPD). Both untreated patients and those being treated with an approved therapy for Pompe disease are eligible to participate. The objectives of the registry are: * To evaluate the long-term safety of Pompe disease treatments through collection of data that describe the frequency of adverse events (AEs)/serious adverse events (SAEs) occurring in Pompe disease patients * To evaluate the long-term real-world effectiveness of Pompe disease treatments * To evaluate the long-term real-world impact of Pompe disease treatments on quality of life (QOL) and patient-reported outcomes (PROs) * To describe the natural history of untreated Pompe disease

Jenel Facey - jenel.facey@cchmc.org

ALL
This study is NOT accepting healthy volunteers
NCT06121011
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Inclusion Criteria:
* Diagnosis of LOPD or IOPD based on documented deficiency of GAA enzyme activity and/or GAA genotyping
Exclusion Criteria:
* Patients who are currently receiving investigational therapy for Pompe disease in a clinical trial, a compassionate use program, or an expanded access program (EAP)
BIOLOGICAL: Cipaglucosidase alfa, DRUG: Miglustat, BIOLOGICAL: Alglucosidase alfa or Avalglucosidase alfa, OTHER: Untreated
Pompe Disease
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Project: Every Child for Younger Patients With Cancer

This study gathers health information for the Project: Every Child for younger patients with cancer. Gathering health information over time from younger patients with cancer may help doctors find better methods of treatment and on-going care.

- cancer@cchmc.org

ALL
Up to 25 years old
This study is NOT accepting healthy volunteers
NCT02402244
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Inclusion Criteria:
* Enrollment must occur within 6 months of initial disease presentation OR within 6 months of refractory disease, disease progression, disease recurrence, second or secondary malignancy, or post-mortem * Patients previously enrolled on ACCRN07 are eligible to enroll on Tracking Outcome, Registry and Future Contact components of APEC14B1 any time after they reach age of majority * Patients with a known or suspected neoplasm that occurs in the pediatric, adolescent or young adult populations are eligible for enrollment as follows: * All cancer cases with an International Classification of Diseases for Oncology (ICD-O) histologic behavior code of one "1" (borderline), two "2" (carcinoma in situ) or three "3" (malignant) * All neoplastic lesions of the central nervous system regardless of behavior, i.e., benign, borderline or malignant * All neoplastic lesions of the kidney regardless of behavior, i.e., benign, borderline or malignant * The following other benign/borderline conditions: * Mesoblastic nephroma * Teratomas (mature and immature types) * Myeloproliferative diseases including transient myeloproliferative disease * Langerhans cell histiocytosis * Lymphoproliferative diseases * Desmoid tumors * Gonadal stromal cell tumors * Neuroendocrine tumors including pheochromocytoma * Melanocytic tumors, except clearly benign nevi * Ganglioneuromas * Subjects must be =\< 25 years of age at time of original diagnosis, except for patients who are being screened specifically for eligibility onto a COG (or COG participating National Clinical Trials Network \[NCTN\]) therapeutic study, for which there is a higher upper age limit * All patients or their parents or legally authorized representatives must sign a written informed consent and agree to participate in at least one component of the study; parents will be asked to sign a separate consent for their own biospecimen submission * If patients or their parents or legally authorized representatives have not signed the Part A subject consent form at the time of a diagnostic bone marrow procedure, it is recommended that they initially provide consent for drawing extra bone marrow using the Consent for Collection of Additional Bone Marrow; consent using the Part A subject consent form must be provided prior to any other procedures for eligibility screening or banking under APEC14B1
OTHER: Cytology Specimen Collection Procedure, OTHER: Medical Chart Review
Adrenal Gland Pheochromocytoma, Carcinoma In Situ, Central Nervous System Neoplasm, Childhood Immature Teratoma, Childhood Kidney Neoplasm, Childhood Langerhans Cell Histiocytosis, Childhood Mature Teratoma, Congenital Mesoblastic Nephroma, Desmoid Fibromatosis, Ganglioneuroma, Lymphoproliferative Disorder, Malignant Neoplasm, Malignant Solid Neoplasm, Melanocytic Neoplasm, Myeloproliferative Neoplasm, Neoplasm of Uncertain Malignant Potential, Neuroendocrine Neoplasm, Stromal Neoplasm
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Registry for Stage 2 Type 1 Diabetes

Stage 2 Type 1 Diabates (T1D) is an early stage of T1D characterized by dysglycemia but not yet leading to clinical symptoms. Progression of the disease to Stage 3 (clinical T1D), leads to overt hyperglycemia requiring eventually exogenous insulin. TZIELD® (teplizumab-mzwv) has been approved to delay onset of stage 3 T1D, by the United States (US) Food and Drug Administration (FDA) for adults and children aged 8 years and older with Stage 2 T1D. The purpose of this study is to collect general information on patients with stage 2 T1D and further information on the long-term effects of TZIELD® in patients with Stage 2 T1D, treated as per standard of care.

Cierra Farrell - cierra.farrell@cchmc.org

ALL
This study is NOT accepting healthy volunteers
NCT06481904
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Inclusion Criteria:
TZIELD-Exposed Cohort * Patients in the US diagnosed with Stage 2 T1D who are planned to initiate TZIELD treatment according to the currently approved label or who have initiated TZIELD treatment within 6 months prior to enrollment: * Day 1: 65 mcg/m2 * Day 2: 125 mcg/m2 * Day 3: 250 mcg/m2 * Day 4: 500 mcg/m2 * Days 5 through 14: 1,030 mcg/m2 per day * Cumulative dose is approximately 11,240 mcg/m2 * Appropriate written informed consent/assent as applicable for the age of the patient TZIELD-Unexposed Cohort * Patients in the US diagnosed with Stage 2 T1D but who are not treated with TZIELD * Appropriate written informed consent/assent as applicable for the age of the patient
Exclusion Criteria:
* Patients who initiated TZIELD treatment more than 6 months prior to enrollment * Patients who had participated in a previous clinical trial for TZIELD * Patients in an ongoing clinical trial of an investigational product or who had ended participation within 6 months prior to study enrollment; patients participating in other observational studies may be enrolled The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
DRUG: TZIELD (teplizumab-mzwv)
Type 1 Diabetes
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Open-Label Study of mRNA-3927 in Participants With Propionic Acidemia

This 3-part, Phase 1/2 study is designed to characterize the safety, tolerability, and pharmacological activity (as assessed by biomarker measurements) and to determine the selected dose of mRNA-3927 in participants with genetically confirmed propionic acidemia (PA). After establishing a dose with an acceptable safety and pharmacodynamic (PD) response for participants ≥1 year of age in Part 1, participants will be enrolled in Part 2 (which will serve as the pivotal study) to allow for determination of the efficacy, safety, and PD of mRNA-3927. Part 3 will evaluate the safety, efficacy and PD response of mRNA-3927 in infants (\<1 year of age).

Laurie Bailey - laurie.bailey@cchmc.org

ALL
PHASE1
This study is NOT accepting healthy volunteers
NCT04159103
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Inclusion Criteria:
Participants ≥1 year of age are eligible to be included in the study only if all of the following criteria apply: * ≥ 8 years of age at the time of consent/assent if enrolled as 1 of the first 2 participants in Part 1. * ≥1 year of age at the time of consent/assent if enrolled after the first 2 participants in Part 1. * Confirmed diagnosis of PA based on diagnosis by molecular genetic testing via central laboratory (PCCA and/or PCCB mutations). * Part 2 only: At least one documented MDE in the 12-month period before consent. Participants \<1 Year of Age : * Identification by newborn screening shortly after birth or having suspected PA by presenting with a spectrum of metabolic symptoms, and having a sibling diagnosed with PA. Participant may enter the Screening Period while awaiting genetic testing results, provided that all other eligibility criteria are met but would not be enrolled until diagnosis of PA is confirmed. * For infants in the neonatal intensive care unit (NICU) only: ≥37 weeks gestational age at the time of birth without other conditions/comorbidities that in the opinion of the Investigator may interfere with the interpretation of study results. * Body weight ≥3 kilograms (kg) at Screening. * At least 1 documented PA-related event prior to Screening defined as the following criteria: * Clinical signs of metabolic deterioration consistent with PA (for example, vomiting, not feeding well/poor suck, heavy breathing, lethargy, absence of proper perfusion, abnormal movements including bicycling, abnormal tone, low body temperature, seizure\[s\]), OR * Meeting the criteria of MDE definition, OR * Evidence of laboratory abnormalities as evidenced by at least one of the following: * Metabolic acidosis with elevated anion gap. * Acute hyperammonemia. * Neutropenia or thrombocytopenia.
Exclusion Criteria:
Participants of all ages are excluded from the study if during Screening any of the following criteria apply: * Any individual with laboratory abnormalities considered to be clinically significant (for example, markedly out of range, associated with clinical symptoms) in the Investigator or Sponsor's opinion that could interfere with or limit the participation in the study. * Estimated glomerular filtration rate (eGFR) \<30 milliliters (mL)/minute/1.73 square meter (m\^2) for participants of all ages receiving chronic dialysis. * History of organ transplantation or planned organ transplantation during the period of study participation. * Corrected QT interval (QTc) \>480 milliseconds (ms) using Bazett's correction. * Grade 3 or 4 heart failure according to the Modified Ross Heart Failure Classification for Children or the New York Heart Association Classification. * Pregnant or breastfeeding. * Other clinically significant conditions that in the Investigator's opinion could interfere with the safety of the participant, the interpretation of study results, or limit the participation in the study.
BIOLOGICAL: mRNA-3927
Propionic Acidemia
mRNA-3927, Propionic Aciduria, Metabolism, Inborn Errors, Genetic Diseases, Inborn Amino Acid Metabolism, Inborn Errors, Acidosis, Acid-Base Imbalance, Metabolic Diseases, Organic Acidemias, Moderna, mRNA
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A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)

This study is an access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs) in pediatric and adult patients with hematologic malignancies and other indications.

513-636-2799 - cancer@cchmc.org

ALL
This study is NOT accepting healthy volunteers
NCT01351545
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Inclusion Criteria:
* Disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment * Signed informed consent (and signed assent, if applicable) obtained prior to study enrollment * Pediatric and adult patients of any age
Exclusion Criteria:
* Patients who are receiving only licensed CBUs * Cord blood transplant recipients at international transplant centers * Patients who are enrolled on another IND protocol to access the unlicensed CBU(s) * Patients whose selected unlicensed CBU(s) will be more than minimally manipulated
DRUG: A multicenter access and distribution protocol for unlicensed cryopreserved cord blood units (CBUs)
Hematologic Malignancies, Inherited Disorders of Metabolism, Inherited Abnormalities of Platelets, Histiocytic Disorders, Acute Myelogenous Leukemia (AML or ANLL), Acute Lymphoblastic Leukemia (ALL), Other Acute Leukemia, Chronic Myelogenous Leukemia (CML), Myelodysplastic (MDS) / Myeloproliferative (MPN) Diseases, Other Leukemia, Hodgkin Lymphoma, Non-hodgkin Lymphoma, Multiple Myeloma/ Plasma Cell Disorder (PCD), Inherited Abnormalities of Erythrocyte Differentiation or Function, Disorders of the Immune System, Autoimmune Diseases, Severe Aplastic Anemia
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A Phase 2 Study of Vosoritide in Children With Idiopathic Short Stature

The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

Leah Tyzinksi - Leah.Tyzinski@cchmc.org

ALL
3 years to 11 years old
PHASE2
This study is NOT accepting healthy volunteers
NCT06382155
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Key
Inclusion Criteria:

• Height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth charts
• If participant is ≥ 5 years at Screening,must be Tanner Stage I to be eligible for enrollment and randomization3. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (≥ -1.00 SDs and ≤+2.00 SDs). Key Exclusions:
• Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome), ACAN deficiency.
• Previous treatment with a growth promoting agent
DRUG: Vosoritide Injection, DRUG: Human Growth Hormone, DRUG: Placebo
Idiopathic Short Stature
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Inspiring New Science In Guiding Healthcare in Turner Syndrome Registry (INSIGHTS)

INSIGHTS is a registry research study that collects key information on medical history for girls and women with Turner syndrome and the clinical care they receive. This includes genetic tests, imaging, medications, and more for hundreds of patients seen at a number of clinics across the US. In addition to learning a lot about the current state of health for individuals with TS, INSIGHTS serves as an infrastructure to conduct future studies are meaningful to patients and their families.

Leah Tyzinksi - leah.tyzinski@cchmc.org

FEMALE
This study is NOT accepting healthy volunteers
NCT05052606
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Inclusion Criteria:

• Individuals with TS and TS variants as defined by the TS Clinical Practice Guideline definition (karyotype consistent with TS, phenotypic female, clinical feature(s) of TS)
• Informed consent/assent as appropriate
Exclusion Criteria:
a. Lack of a TS diagnosis on file
OTHER: No intervention
Turner Syndrome
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A Study to Investigate Efficacy and Safety of Teplizumab Compared With Placebo in Participants 1 to 25 Years of Age With Stage 3 Type 1 Diabetes (βETA PRESERVE)

This is a multicenter, randomized, double-blind, parallel, placebo-controlled Phase 3, 2-arm study for treatment. The purpose of this study is to measure change in glycemic control and prandial insulin independency over 52 weeks with teplizumab compared with placebo, both administered by intravenous (IV) infusion, in participants with recently diagnosed Stage 3 type 1 diabetes (T1D) aged 1 to 25 years, on standard insulin therapy.

Cierra Farrell - cierra.farrell@cchmc.org

ALL
1 year to 25 years old
PHASE3
This study is NOT accepting healthy volunteers
NCT07088068
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Inclusion Criteria:
* Participants are eligible to be included in the study only if all of the following criteria apply: * Participant must be 1 to 25 years of age inclusive, at the time of signing the informed consent. * Participants diagnosed with T1D Stage 3 according to American Diabetes Association 2025 criteria * Participants able to be randomized and initiate study drug within 8 weeks (56 days) of the Stage 3 T1D diagnosis * Participants must be positive for at least one T1D autoantibody at screening: * Glutamic acid decarboxylase (GAD-65), * Insulinoma Antigen-2 (IA-2), * Zinc-transporter 8 (ZnT8), or * Insulin (if obtained not later than 14 days after exogenous insulin therapy initiation). * Islet cell cytoplasmic autoantibodies (ICAs) * Have random C-peptide level ≥0.2 nmol/L obtained at screening * Enter Inclusion Criteria Sex * Both male and female participants are eligible. * Contraceptive use by women should be consistent with local regulations regarding the methods of contraception for those participating in clinical studies. * A female participant is eligible to participate if she is not pregnant, and one of the following conditions applies: * Is a woman of nonchildbearing potential (WONCBP) OR * Is a woman of childbearing potential (WOCBP) and agrees to use a contraceptive method that is highly effective, with a failure rate of \<1% during the study intervention period (to be effective before starting the intervention) and for at least 30 days after the last administration of study intervention. * A WOCBP must have a negative highly sensitive pregnancy test at screening (serum) and within 24 hours (urine or serum as required by local regulations) before the first administration of study intervention. * Lactating woman must interrupt breastfeeding and pump and discard breast milk during and for 20 days after last administration of study intervention. * Capable of giving signed informed consent as described in Appendix 1 of the protocol which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. Note: For minor participants, a specific ICF must also be signed by the participant's legally authorized representative (LAR).
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply: * Participant has diabetes other than autoimmune T1D that includes but is not limited to genetic forms of diabetes, maturity-onset diabetes of the young (MODY), diabetes secondary to medications or surgery and type 2 diabetes by judgement of the Investigator. * Participant has an active serious infection and/or fever ≥38.5°C (101.3°F) within the 48 hours prior to the first dose (except if localized skin infection), or has chronic, recurrent or opportunistic infectious disease. * At screening, participant has laboratory or clinical evidence of acute or clinically active infection with Epstein-Barr virus (EBV), cytomegalovirus (CMV). * At screening, participant has positive serology for human immunodeficiency virus (HIV), hepatitis B (HBV), or hepatitis C (HCV). * Participant has evidence of active or latent tuberculosis (TB) as documented by medical history and examination, chest X-rays (posterior anterior and lateral), and/or TB testing. Blood testing (eg, QuantiFERON® TB Gold test) is strongly preferred; if not available, any local approved TB test is allowed. * Has other autoimmune diseases, (eg, rheumatoid arthritis, polyarticular juvenile idiopathic arthritis, psoriatic arthritis, ankylosing spondylitis, multiple sclerosis, systemic lupus erythematosus etc), except clinically stable autoimmune thyroid disease, or controlled celiac disease (at discretion of Investigator). * Any clinically significant abnormality identified either in medical/surgical history or during screening evaluation (eg, physical examination, laboratory tests, vital signs), or any adverse event (AE) during screening period which, in the judgment of the investigator, would preclude safe completion of the study or constrains efficacy assessment. * Participant has recent or planned vaccinations as follows: * Live-attenuated (live) vaccines (eg, varicella, measles, mumps, rubella, cold-attenuated intranasal influenza vaccine, and smallpox) within the 8 weeks before first dose of the investigational medicinal product (IMP) or planned/required administration during treatment or up to 26 weeks after last IMP administration in any treatment course * Inactivated or mRNA vaccines within 2 weeks before the first dose of IMP or planned required administration during treatment or up to 6 weeks after last IMP administration in any treatment course. * Current or prior use (within 30 days before screening) of any anti-hyperglycemic agents other than insulin * Past (within 30 days prior to screening) or current administration of any treatment that is known to cause a significant, ongoing change in the course of T1D or immunologic status, including but not limited to systemic corticosteroids (ie, oral, high doses of inhaled or injectable) with duration \>14 days, adrenocorticotropic hormone, verapamil). * Past systemic immunosuppression medicine or immune modulatory biologic therapy (such as monoclonal antibodies), within 3 months or 5 half-lifes (whichever is longer) prior to dosing. * Current or prior (within 30 days before screening) use of any medication known to significantly influence glucose tolerance (eg, atypical antipsychotics, diphenylhydantoin, niacin). * Participant has previously received teplizumab or other anti-CD3 treatment. * Other medications not compatible or interfering with IMP at discretion of Investigator. * Current enrollment OR past participation in another investigational study in which an investigational intervention (eg, drug, vaccine, invasive device) was administered within the last 8 weeks or 5 half-lifes, whichever is longer, prior to screening. * Participant has any of the following laboratory parameters, at screening prior to first dose: * Lymphocyte count: \<1000/µL, * Neutrophil count: \<1500/µL (\< 1000 /µL in participants with documented Duffy-null genotype), * Platelet count: \<150,000 platelets/µL, * Hemoglobin: \<10 g/dL, * Aspartate aminotransferase (AST) \>2.0 × upper limit of normal (ULN), * Alanine aminotransferase (ALT) \>2.0 × ULN, * Total bilirubin \>1.5 × ULN with the exception of participants with the diagnosis of Gilbert's syndrome who may be eligible provided they have no other causes leading to hyperbilirubinemia The above information is not intended to contain all considerations relevant to a participant's potential participation in a clinical trial.
DRUG: Teplizumab, OTHER: Placebo
Type 1 Diabetes Mellitus
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A Study to Evaluate the Safety, Efficacy, PK, PD and Immunogenicity of Cipaglucosidase Alfa/Miglustat in IOPD Subjects Aged 0 to <18 (ROSSELLA)

This is a Phase 3, open-label, multicenter study to evaluate the safety, efficacy, PK, PD, and immunogenicity of cipaglucosidase alfa/miglustat treatment in ERT-experienced and ERT-naïve pediatric subjects with IOPD.

Laurie Bailey - laurie.bailey@cchmc.org

ALL
Up to 17 years old
PHASE3
This study is NOT accepting healthy volunteers
NCT04808505
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Inclusion Criteria:
Cohort 1:
• Male or female subjects who are aged 6 months to \< 18 years on Day 1
• Subject must have documentation of IOPD genotype
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject must have received ERT for at least 6 months immediately before enrollment. For subjects whose ERT dosage has been modified, the subject must have been on the modified dosage and regimen for at least 3 months before enrollment
• Subjects aged ≥ 12 to \< 18 years must perform one valid 6-minute walk test (6MWT) (≥ 75 meters) at screening; Subjects aged ≥ 5 to \< 12 years must perform one valid 6MWT (≥ 40 meters) at screening; Subjects aged 18 months to \< 5 years must be ambulatory and assessed to be likely to be able to perform 6MWT (≥ 40 meters) when they turn 5 years old
• Subjects must have experienced a clinical decline on their current rhGAA dose and frequency Cohort 2:
• Male or female subjects who are aged 0 to \<6 months at Day 1
• Subject must have documentation of IOPD genotype
• Subject must have had hypertrophic cardiomyopathy at the time of diagnosis
• Subject is ERT-naïve Long-term Extension (Cohort 1 or Cohort 2): 1\. Subject must have, in the opinion of the investigator, benefited from therapy with cipaglucosidase alfa/miglustat during the 104-week primary treatment period with no significant safety concerns.
Exclusion Criteria:
Cohort 1 and Cohort 2, unless specified
• Subject requires invasive ventilation (eg, tracheostomy)
• Subject is CRIM negative and has not received prophylactic immunomodulation (Cohort 1); Subject is CRIM negative and will not be receiving prophylactic immunomodulation (Cohort 2)
• Subject has a history of life-threatening IARs/hypersensitivity (eg, anaphylaxis and severe cutaneous reactions) to ERT (eg, alglucosidase alfa, cipaglucosidase alfa, miglustat) or other iminosugars, or to any of the excipients, where rechallenge was unsuccessful
• Subject has prior history of illness or condition known to affect motor function
• Female subject is pregnant (or intends to get pregnant) or breastfeeding at screening (Cohort 1)
BIOLOGICAL: Cipaglucosidase alfa, DRUG: Miglustat
Glycogen Storage Disease Type II Infantile Onset
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